EP4194553A4 - MODIFIED SIRNA WITH REDUCED OFF-TARGET ACTIVITY - Google Patents

MODIFIED SIRNA WITH REDUCED OFF-TARGET ACTIVITY Download PDF

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Publication number
EP4194553A4
EP4194553A4 EP21852482.5A EP21852482A EP4194553A4 EP 4194553 A4 EP4194553 A4 EP 4194553A4 EP 21852482 A EP21852482 A EP 21852482A EP 4194553 A4 EP4194553 A4 EP 4194553A4
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EP
European Patent Office
Prior art keywords
reduced
target activity
modified sirna
sirna
modified
Prior art date
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Pending
Application number
EP21852482.5A
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German (de)
English (en)
French (fr)
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EP4194553A1 (en
Inventor
Jinyu Huang
Min Luo
Ke YIN
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Tuojie Biotech Shanghai Co Ltd
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Tuojie Biotech Shanghai Co Ltd
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Publication of EP4194553A1 publication Critical patent/EP4194553A1/en
Publication of EP4194553A4 publication Critical patent/EP4194553A4/en
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1131—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1136—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against growth factors, growth regulators, cytokines, lymphokines or hormones
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/313—Phosphorodithioates
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/319—Chemical structure of the backbone linked by 2'-5' linkages, i.e. having a free 3'-position
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/3235—Chemical structure of the sugar modified ring structure having the O of the ribose replaced by another atom
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
EP21852482.5A 2020-08-04 2021-08-04 MODIFIED SIRNA WITH REDUCED OFF-TARGET ACTIVITY Pending EP4194553A4 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
CN202010772542 2020-08-04
CN202110244977 2021-03-05
CN202110361502 2021-04-02
PCT/CN2021/110509 WO2022028462A1 (zh) 2020-08-04 2021-08-04 脱靶活性降低的修饰sirna

Publications (2)

Publication Number Publication Date
EP4194553A1 EP4194553A1 (en) 2023-06-14
EP4194553A4 true EP4194553A4 (en) 2024-11-13

Family

ID=80120017

Family Applications (1)

Application Number Title Priority Date Filing Date
EP21852482.5A Pending EP4194553A4 (en) 2020-08-04 2021-08-04 MODIFIED SIRNA WITH REDUCED OFF-TARGET ACTIVITY

Country Status (10)

Country Link
US (1) US20230287418A1 (https=)
EP (1) EP4194553A4 (https=)
JP (1) JP7849353B2 (https=)
KR (1) KR20230043195A (https=)
CN (1) CN115955973A (https=)
BR (1) BR112023002080A2 (https=)
CA (1) CA3190097A1 (https=)
TW (1) TW202214858A (https=)
WO (1) WO2022028462A1 (https=)
ZA (1) ZA202303275B (https=)

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TW202302851A (zh) * 2021-04-02 2023-01-16 大陸商上海拓界生物醫藥科技有限公司 Ｂ型肝炎病毒ｓｉＲＮＡ和ｓｉＲＮＡ綴合物
US20240229037A1 (en) * 2021-04-22 2024-07-11 Tuojie Biotech(Shanghai) Co., Ltd. SIRNA TARGETING 17Beta-HYDROXYSTEROID DEHYDROGENASE TYPE 13 AND SIRNA CONJUGATE
WO2023109932A1 (zh) * 2021-12-16 2023-06-22 上海拓界生物医药科技有限公司 一种dsRNA、其制备方法及应用
US20260076990A1 (en) 2022-01-20 2026-03-19 Tuojie Biotech (Shanghai) Co., Ltd. Dsrna, use thereof and preparation method therefor
KR20250123805A (ko) * 2022-12-13 2025-08-18 투오지에 바이오텍 (상하이) 컴퍼니 리미티드 친유성 단량체를 포함하는 올리고뉴클레오티드 및 비-간 전달에서의 용도
JP2026503329A (ja) * 2023-02-21 2026-01-28 成都先導薬物開発股▲フン▼有限公司 ヌクレオチド類似体、その調製方法及び使用
EP4674965A1 (en) 2023-03-02 2026-01-07 Tuojie Biotech (Shanghai) Co., Ltd. Sirna targeting inhibin ?e, sirna conjugate, and medical use thereof
WO2024217152A2 (zh) * 2023-04-21 2024-10-24 苏州时安生物技术有限公司 一种寡聚核酸及其制备方法和应用
AU2024262610A1 (en) 2023-04-27 2025-10-16 Jiangsu Hengrui Pharmaceuticals Co., Ltd. Intermediate for preparing oligonucleotide analog or salt thereof, and preparation method therefor
CN121079106A (zh) * 2023-05-31 2025-12-05 上海拓界生物医药科技有限公司 一种靶向LPA的dsRNA及其应用
AU2024302964A1 (en) 2023-06-13 2025-12-18 Tuojie Biotech (Shanghai) Co., Ltd. Sirna targeting mmp7, sirna conjugate and medical use thereof
WO2025007907A1 (zh) * 2023-07-04 2025-01-09 大睿生物 包含肽的寡核苷酸递送配体
AU2024315297A1 (en) 2023-08-01 2026-02-12 Tuojie Biotech (Shanghai) Co., Ltd. Dsrna targeting mapt and pharmaceutical use thereof
CN121586578A (zh) 2023-08-24 2026-02-27 上海拓界生物医药科技有限公司 靶向RAGE的RNAi剂及其医药用途
TW202526017A (zh) * 2023-09-14 2025-07-01 美商Ionis製藥公司 用於減少ａｐｏｃｉｉｉ表現的化合物及方法
WO2025067317A1 (zh) * 2023-09-26 2025-04-03 上海拓界生物医药科技有限公司 一种脂质修饰的核酸化合物、其制备方法及用途
WO2025140299A1 (zh) * 2023-12-26 2025-07-03 广东东阳光药业股份有限公司 一种新型的化合物及其用途
WO2026002138A1 (zh) * 2024-06-27 2026-01-02 成都新泽利医药科技有限公司 特定修饰的双链寡核苷酸及其组合物
CN119410644A (zh) * 2025-01-08 2025-02-11 北京安龙生物医药有限公司 修饰的双链寡核苷酸及其缀合物和组合物
CN119528986B (zh) * 2025-01-23 2025-09-16 北京悦康科创医药科技股份有限公司 一种核苷类似物及其在降低寡核苷酸药物脱靶活性中的应用

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